Verastem

Description: This presentation and other matters discussed today, or answers that may be given today, include forward-looking statements about Verastem’s strategy, future plans and prospects, including statements regarding the development and activity of Verastem’s investigational product candidates, including duvelisib and defactinib, and Verastem’s PI3K and FAK programs generally, the structure of our planned and pending clinical trials and the timeline and indications for clinical development. The words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements,

FORWARD-LOOKING STATEMENTS
This presentation and other matters discussed today, or answers that may be given today, include forward-looking statements about
Verastem’s strategy, future plans and prospects, including statements regarding the development and activity of Verastem’s
investigational product candidates, including duvelisib an ... See more

defactinib, and Verastem’s PI3K and FAK programs generally, the
structure of our planned and pending clinical trials and the timeline and indications for clinical development. The words “anticipate,”
“believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,”
“continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements
contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results
to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks that the
full data from the DUO study will not be consistent with the top-line results of the study; that the preclinical testing of Verastem’s
product candidates and preliminary or interim data from clinical trials may not be predictive of the results or success of ongoing or
later clinical trials; that even if data from clinical trials is positive, regulatory authorities may require additional studies for approval
and the product may not prove to be safe and effective; that the degree of market acceptance of product candidates, if approved,
may be lower than expected; that the timing, scope, and rate of reimbursement for our product candidates is uncertain; that there
may be competitive developments affecting our product candidates; that data may not be available when we expect it to be; that
enrollment of clinical trials may take longer than expected; that our product candidates will cause unexpected safety events or result
in an unmanageable safety profile as compared to their level of efficacy; that duvelisib will be ineffective at treating patients with
lymphoid malignancies; that Verastem will be unable to successfully initiate or complete the clinical development of its product
candidates; that the development of Verastem’s product candidates will take longer or cost more than planned; that Verastem may
not have sufficient cash to fund its contemplated operations; that Verastem or Infinity Pharmaceuticals, Inc. (Infinity) will fail to fully
perform under the duvelisib license agreement; that Verastem will not pursue or submit regulatory filings for its product candidates;
and that Verastem’s product candidates will not receive regulatory approval, become commercially successful products, or result in
new treatment options being offered to patients. Other risks and uncertainties include those identified under the heading “Risk
Factors” in Verastem’s Annual Report on Form 10-K for the year ended December 31, 2016, and in any subsequent filings with the
U.S. Securities and Exchange Commission. The forward-looking statements contained in this presentation reflect Verastem’s views as
of the date of this presentation, and Verastem does not undertake and specifically disclaims any obligation to update any forwardlooking statements.
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Verastem, Inc.

VERASTEM AT A GLANCE
SCIENTIFIC FOUNDATION

VALUE DRIVERS
Presentation of full DUO data

Novel drugs targeting malignant cells both
directly and through modulation of the
tumor microenvironment

Targeted initiation by EOY 2017
In long term follow up
In long term follow up

Relapsed/Refractory T Cell Lymphoma*
With Romidepsin or Bortezomib

DEFACTINIB (FAK INHIBITOR)
Ovarian
With avelumab

NSCLC, Pancreatic, Mesothelioma*
With pembrolizumab

Pancreatic, relapsed*
With pembrolizumab + gemcitabine
* - Investigator Sponsored Trial (IST)
Duvelisib and defactinib are investigational agents available for clinical trial use only. Safety and efficacy have not been established.

Administered without required hospitalization or infusion center
Manageable safety profile observed to date, well-characterized in >500 patients
Clinical activity observed across B cell and T cell malignancies
IP: COM 2030 before extensions; Orphan Designation: CLL, FL, and SLL in the US and EU
FDA Fast Track Designation: Patients with CLL who have received at least 1 prior therapy; Patients with FL who have received at least 2 prior
therapies; Patients with PTCL who have received at least one prior therapy.

HR = 0.41; p = 0.0011
Duvelisib monotherapy had a manageable safety profile, with results from this study
consistent with the well-characterized safety profile of duvelisib monotherapy observed
to date in patients with advanced hematologic malignancies.
Detailed results to be submitted for peer-reviewed publication
and for presentation at an upcoming scientific meeting
IRC: Independent Review Committee; ITT: Intent-to-Treat
Duvelisib is an investigational agent available for clinical trial use only. Safety and efficacy have not been established.
Verastem, Inc.

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DUO™ MAY OPEN AN INITIAL COMMERCIAL OPPORTUNITY FOR
DUVELISIB IN A GROWING LYMPHOID MALIGNANCY MARKET
CLL MARKET OPPORTUNITY

UNMET NEED REMAINS FOR PATIENTS WITH CLL, THE
MAJORITY OF WHICH PROGRESS FOLLOWING 1L THERAPY
Average CLL patient
72 y.o. male treated outside of
academic institutions, often
with multiple comorbidities1,2,3

“IMBRUVICA may increase the
risk of hemorrhage in patients
receiving antiplatelet or
anticoagulant therapies and
patients should be monitored
for signs of bleeding.”
“Atrial fibrillation and atrial
flutter (range, 6 to 9%) have
occurred in patients treated
with IMBRUVICA, particularly in
patients with cardiac risk
factors, hypertension, acute
infections, and a previous
history of atrial fibrillation.”

A subset of patients are not eligible for
subs
or intolerant to BTK inhibitors due to
co-morbidities or risk factors.

Rituxan®
500 mg/m2

DAILY

I N D I C AT I O N

WARNINGS & PRECAUTIONS

DAILY

Rituxan®

DOSES 2 – 5:
BI-WEEKLY
DOSES 6 – 9:
MONTHLY

“Relapsed chronic lymphocytic
leukemia (CLL), in combination
with rituximab, in patients for
whom rituximab alone would be
considered appropriate therapy
due to other co-morbidities.”

Zydelig® is approved only in
combination with IV rituximab,
increasing the cost and complexity of
treatment for patients.

Verastem will pay to Infinity up to $28 million in milestones
− First milestone of $6 million payable upon positive data from the DUO study
− $22 million milestone payable upon the first regulatory approval in any territory

•

Milestones are payable in cash or equity at Verastem’s option

•

Verastem to pay tiered high single to low double digit royalties on net sales